Originally created 08/14/97

Lou Gehrig's patients take to streets in push for drug

WASHINGTON (AP) - Chris Pendergast paced outside the Food and Drug Administration holding photographs of fellow sufferers from Lou Gehrig's disease. Suddenly, his weakened legs collapsed and he rolled into the busy highway.

Shaken, the Long Island, N.Y., man struggled to his feet and promised that his tiny band of protesters, many partially paralyzed and in wheelchairs, would continue pushing the government to approve an unproven therapy for their devastating disease.

"One day I'm going to be chained to that door," Pendergast threatened, pointing to where FDA scientists debated the drug called Myotrophin. "It's the politics of power, and we have been powerless for too long."

In a classic clash of emotion with science, feeble Lou Gehrig's patients have begun copying politically powerful AIDS activists - posting banners of victims' photographs and flooding FDA officials with phone calls and electronic mail. Two thousand e-mails were sent Sunday night alone.

They hope the pressure of desperate people will spur the FDA to approve Myotrophin, a drug that so far lacks clear proof that it helps.

"If anybody thinks people here don't know this is a horrible, horrible disease, don't want to reach out and hug the people and do something about it, they're wrong," said Dr. Robert Temple, the FDA drug chief who will decide - by Nov. 11 - if Myotrophin provides enough evidence of possible benefit to let it sell.

But "it's not a favor to people to give them something that doesn't work," Temple said.

Temple disputes patients' complaints that Lou Gehrig's drugs are held to a stricter standard than AIDS medications. He's frustrated with wading through protesters' e-mail to get to the scientific correspondence that will help his decision. "They're getting on my nerves," he admits.

Some 30,000 Americans suffer Lou Gehrig's disease, formally known as amyotrophic lateral sclerosis or ALS. It destroys nerve cells in the brain and spinal cord, leaving patients prisoners inside progressively paralyzed bodies and killing them in three to five years. There is one treatment, Rilutek, but it prolongs some patients' lives only a few months.

Myotrophin is a genetically engineered growth factor thought to help nerve cells survive and thus slow paralysis.

The dilemma: One study in North America suggested Myotrophin might offer about three months of better health but a European study found no benefit - and more of the Europeans died while taking the drug than while taking a dummy pill. Which one does FDA believe?

The Europeans might merely have been sicker to start with, but independent neurologists who advise the FDA told manufacturer Cephalon Inc. a year ago to do a tie-breaking study. Cephalon said it already had spent $180 million developing Myotrophin and wouldn't spend any more, so the scientists urged the FDA in May to reject the drug. The FDA this week postponed its decision, promising to evaluate new Cephalon analyses.

Patients are convinced the drug works, and can't understand why the FDA doesn't approve it on the basis of the North American study alone.

They argue the agency has approved AIDS drugs with such limited data under pressure from AIDS activists who, in the late 1980s, did force the agency to change how it evaluates drugs for untreatable killers.

The government, however, says it doesn't hold ALS treatments to any higher standard. The first AIDS drug, Temple explained, was approved because it had a stunningly obvious effect - only one patient taking AZT died during the trial vs. 16 people who took a dummy pill.

And the first Lou Gehrig's drug, Rilutek, was approved on the basis of two small, foreign studies that were only barely statistically significant, Temple said. Some doctors even accused the FDA of lowering its standards for Rilutek.

"What we do in one disease affects other diseases, if you believe in evidence-based medicine, and we do," Temple said.

But patients say they see evidence the drug works. Outside the FDA last week, Kyle Hahn of Germantown, Md., sat in his wheelchair and demonstrated how, after testing Myotrophin for five months, he regained movement in his hands. He still can hardly talk, but he can swallow solid food again and regained 18 pounds.

Says his friend, Terry Frank: "It bewilders us when Kyle can get up there and show `I have improved' and they won't listen."


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